September 22, 2017 (Medical News Today)
Multiple sclerosis (MS) involves the attack and destruction of myelin around nerve fibers which contribute to a wide range of symptoms. To help protect the myelin in people with MS for the long-term, researchers sought out to develop a gene immunotherapy strategy.
Using an adeno-associated virus (AAV) vector, this approach would deliver a gene encoded with the myelin protein MOG to the liver that would prompt the activity of regulatory T cells which are specific to MOG. These would suppress the destructive activity of self-reactive effector T cells.
This approach was put into action using mice and results found that it prevented the rodents from developing the clinical symptoms associated with MS. The gene therapy also reversed a number of symptoms in the mice who had developed MS.
This approach may not work as well as in humans as it has in mice given that mouse models of MS are far less complex than that in humans, and researchers suspect that the therapy will not be available to patients with MS for at least five years.
Published in the journal Molecular Therapy.